About End Alexander Disease
End Alexander Disease, Inc. (End AxD) is a 501(c)(3) nonprofit working to change the future for families affected by Alexander Disease — a rare, progressive condition that has long lacked dedicated research, treatments, and coordinated community support.
We are a community-led organization. Every member of our board has a personal family connection to Alexander Disease. That's not incidental — it's the foundation of how we operate and how we make decisions.
Our Mission
“End AxD is fiercely committed to serving the Alexander Disease community by accelerating research into treatments and a cure, advocating for, supporting, and empowering patients and families.”
Our Vision
A world where Alexander's Disease no longer limits lives, where families are empowered with knowledge, connection, and hope, and where transformative research leads to treatments and hopefully a cure bringing real change to those impacted by this rare condition.
At End AxD, we envision a future where:
Every individual affected by Alexander's Disease has access to cutting-edge treatments and comprehensive care.
Families are equipped with the resources, support, and community they need to feel empowered through their journey.
The broader public is informed about Alexander's Disease, fostering awareness and understanding.
Advocacy efforts drive policy changes, ensuring equitable research funding and healthcare support.
Collaborative partnerships accelerate groundbreaking research, uncovering cures and interventions that redefine the outlook for Alexander's Disease.
Our commitment to research, connection, advocacy, and hope fuels our determination to make this vision a reality, one step at a time.
How We Work
Our work is organized around interconnected areas, each led by board members who personally drive it forward:
Patient & Family Support — Connecting families to community, resources, and one another. Helping navigate care, diagnosis, and the long road that follows.
Research & Drug Development — Investing in the science. Supporting clinical research at the Children's Hospital of Philadelphia (CHOP), advancing the Ionis 373 (Zilganersen) program, and identifying future treatments through our Scientific Advisory Committee.
Awareness & Advocacy — Putting Alexander Disease on the map. The EL-PFDD initiative, awareness campaigns, and outreach to the broader rare-disease community.
Fundraising — Building the financial foundation that makes the rest possible. Monthly giving, events, partnerships, and the everyday work of resourcing the mission.
A Word About Capacity
Our board is small. Every member balances End AxD with the rest of their lives — including the day-to-day reality of being personally impacted by Alexander Disease. We are honest about what we can do today and what we're building toward. We are not a large, well-resourced organization — we are a determined community.